FDA reviewers on Ipsen’s potential ultra-rare disease drug: Post-hoc analysis may be enough

Post-hoc analy­ses are gen­er­al­ly a ma­jor no-no for the FDA, but agency re­view­ers sig­naled their open­ness to con­sid­er­ing them in the case of Ipsen’s po­ten­tial new ther­a­py for pa­tients with an ul­tra-rare ge­net­ic con­nec­tive tis­sue dis­or­der.

The FDA’s En­docrino­log­ic and Meta­bol­ic Drugs Ad­vi­so­ry Com­mit­tee of out­side ex­perts will meet Wednes­day to dis­cuss whether it is rea­son­able to con­clude that the drug, known as palo­varotene, is safe and ef­fec­tive for pa­tients with fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va. The pan­elists will re­view one Phase II study with mul­ti­ple open-la­bel ex­ten­sion phas­es and one sin­gle-arm, open-la­bel Phase III study with a nat­ur­al his­to­ry study as the ex­ter­nal con­trol to sup­port the ef­fi­ca­cy of palo­varotene. Ipsen is re­quest­ing ap­proval of 5 mg dai­ly, with flare-up dos­ing at 20 mg dai­ly for 4 weeks, fol­lowed by 10 mg dai­ly for 8 weeks.

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